A Multicenter, Phase 4, Randomized, Controlled Study to Evaluate the Efficacy and Safety of Recombinant Alpha-Galactosidase A (Agalsidase Beta, FABRAZYME) in Heterozygous Females for Fabry Disease
试验速览
- 阶段
- 4 期
- 入组人数
- 34
- 试验地点
- 1
- 主要终点
- Left ventricular mass
研究概览
简要总结
Fabry disease (OMIM 301500) is an X-linked inborn error of sphingolipid metabolism resulting from the deficiency of the lysosomal enzyme alpha-galactosidase A. Heterozygous females for Fabry disease may be symptomatic with cardiac, renal or cerebrovascular involvement. Clearance of Gb3 and stabilization of renal function has been demonstrated in male patients treated with agalsidase beta (FABRAZYME). In contrast, no randomized, controlled study of the efficacy of recombinant alpha-galactosidase A has been reported in heterozygotes for Fabry disease.
详细描述
The primary objective is to evaluate cardiac left ventricular mass (measured with echocardiography by unique investigator) in females over 15 years of age affected with Fabry disease receiving 70 mg of agalsidase beta every other week, as compared with an untreated controlled group matched for gender and age.
The secondary objectives include evaluation of :
- left ventricular posterior wall thickness (echocardiography)
- interventricular septum thickness (echocardiography)
- tissue doppler imaging (myocardial function)
- EKG
- creatinaemia
- serum cystatin C level
- urinary protein/creatinine ratio
- microalbuminuria
- Gb3 urinary levels
Evaluation of tolerance and safety with :
- Home therapy infusions follow up
- Vitals
- Physical examination
- Adverse events
- Antibodies levels
研究设计
- 研究类型
- Interventional
- 分配方式
- Randomized
- 干预模型
- Parallel
- 主要目的
- Treatment
- 盲法
- None
入排标准
- 年龄范围
- 15 Years 至 —（Child, Adult, Older Adult）
- 性别
- Female
- 接受健康志愿者
- 否
入选标准
- •Female patients over 15 years with clinical and biological evidence of Fabry disease (GLA gene mutation detected)
排除标准
- •Pregnancy
- •Allergy to agalsidase beta
- •Congestive heart failure
- •Creatinaemia > 135 µmol/l
- •Medical history of stroke during the last year
- •Medical history of more than 2 transient ischemic attack
- •Blood pressure > 160/95
- •Modification in medications treating for blood pressure during the last 3 months before enrollment
- •Complete absence of clinical or biological symptoms
- •Weight > 87 kg or < 35 kg
结局指标
主要结局
Left ventricular mass
时间窗: 2 years
次要结局
- Posterior wall thickness, interventricular thickness, ECG, creatinaemia, urinary protein / creatinine ratio, microalbuminuria, urinary Gb3 level(2 years)